CINXE.COM
Our Therapies | Clinical information about Alnylam’s RNAi-based therapies
<!DOCTYPE html> <html lang="en" dir="ltr" prefix="content: http://purl.org/rss/1.0/modules/content/ dc: http://purl.org/dc/terms/ foaf: http://xmlns.com/foaf/0.1/ og: http://ogp.me/ns# rdfs: http://www.w3.org/2000/01/rdf-schema# schema: http://schema.org/ sioc: http://rdfs.org/sioc/ns# sioct: http://rdfs.org/sioc/types# skos: http://www.w3.org/2004/02/skos/core# xsd: http://www.w3.org/2001/XMLSchema# "> <head> <meta charset="utf-8" /> <meta name="description" content="Explore resources related to the FDA-approved uses of Alnylam therapies in transthyretin (ATTR) amyloidosis, acute hepatic porphyria (AHP), and primary hyperoxaluria type 1 (PH1)." /> <link rel="canonical" href="https://www.rnaiscience.com/our-therapies" /> <meta property="og:url" content="https://www.rnaiscience.com/our-therapies" /> <meta property="og:title" content="Our Therapies | Clinical information about Alnylam’s RNAi-based therapies" /> <meta property="og:description" content="Explore resources related to the FDA-approved uses of our therapies" /> <meta property="og:image" content="https://www.rnaiscience.com/sites/default/files/2024-02/degrading-mrna.png" /> <meta name="Generator" content="Drupal 10 (https://www.drupal.org)" /> <meta name="MobileOptimized" content="width" /> <meta name="HandheldFriendly" content="true" /> <meta name="viewport" content="width=device-width, initial-scale=1.0" /> <link rel="icon" href="/sites/default/files/al-favicon.png" type="image/png" /> <title>Our Therapies | Clinical information about Alnylam’s RNAi-based therapies</title> <link rel="stylesheet" media="all" href="/sites/default/files/css/css_qc19X0GuKOFp1QvENBu9cYYXnzfkvfmK_v8Kc34nNCk.css?delta=0&language=en&theme=alnylam&include=eJxVjEEOwyAMBD9Ew5uWYhHUxSA7SCWvz6VN1ePMrNaXH9JigksAdREtFvYEPliTwdat_zZN3FHEw7ObRO3WwHpKkPfBqq-YbQ5w-2AYMBTD2P1bfmabOmZi9V3y_a89ywWWFzzY" /> <link rel="stylesheet" media="all" href="/sites/default/files/css/css_ZaBVkdZE1402zKhuTwGzekLRI7zjO5NNNaP46ohduao.css?delta=1&language=en&theme=alnylam&include=eJxVjEEOwyAMBD9Ew5uWYhHUxSA7SCWvz6VN1ePMrNaXH9JigksAdREtFvYEPliTwdat_zZN3FHEw7ObRO3WwHpKkPfBqq-YbQ5w-2AYMBTD2P1bfmabOmZi9V3y_a89ywWWFzzY" /> <!-- Hotjar Tracking Code for RNAi Science --> <script> (function(h,o,t,j,a,r){ h.hj=h.hj||function(){(h.hj.q=h.hj.q||[]).push(arguments)}; h._hjSettings={hjid:3915815,hjsv:6}; a=o.getElementsByTagName('head')[0]; r=o.createElement('script');r.async=1; r.src=t+h._hjSettings.hjid+j+h._hjSettings.hjsv; a.appendChild(r); })(window,document,'https://static.hotjar.com/c/hotjar-','.js?sv='); </script> <!-- Google Tag Manager --> <script>(function(w,d,s,l,i){w[l]=w[l]||[];w[l].push({'gtm.start': new Date().getTime(),event:'gtm.js'});var f=d.getElementsByTagName(s)[0], j=d.createElement(s),dl=l!='dataLayer'?'&l='+l:'';j.async=true;j.src= 'https://www.googletagmanager.com/gtm.js?id='+i+dl;f.parentNode.insertBefore(j,f); })(window,document,'script','dataLayer','GTM-NLQHVTWH');</script> <!-- End Google Tag Manager --> </head> <body class="path-node page-node-type-generic"> <!-- Google Tag Manager (noscript) --> <noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-NLQHVTWH" height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript> <!-- End Google Tag Manager (noscript) --> <a href="#main-content" class="visually-hidden focusable skip-link"> Skip to main content </a> <div class="dialog-off-canvas-main-canvas" data-off-canvas-main-canvas> <div class="container-xl"> <div class="layout-container row"> <header role="banner" class="container-fluid"> <div class="row"> <nav class="mobile-header col-12 d-block d-xl-none"> <div class="row top"> <div class="utility-menu-wrapper col-12"> <nav class="utility-menu-wrapper"> <ul class="al-menu-utility-menu menu"> <li class="menu-item"> <a href="/connect-with-us#adverse-events-product-complaints" data-drupal-link-system-path="node/24">Report an Adverse Event</a> </li> <li class="menu-item"> <a href="/connect-with-us#msl-locator" data-drupal-link-system-path="node/24">Locate an MSL</a> </li> </ul> </nav> </div> </div> <div class="row bottom"> <div class="left-side col-8"> <a href="/"> <img class="branding" src="/themes/custom/alnylam/assets/dist/images/rnaiscience-logo.svg" alt="RNAiScience logo"/> </a> </div> <div class="right-side col-4"> <a href="/search"> <img class="search-icon" src="/themes/custom/alnylam/assets/dist/images/search.svg" alt="Search" /> </a> <a role="button" class="ham-icon" tabindex="0" href="#"> <img class="ham-icon" src="/themes/custom/alnylam/assets/dist/images/ham.svg" alt="Menu"/> </a> <div class="mobile-menu-expanded d-none"> <div class="menu-wrapper"> <button type="button" class="btn close" tab-index="0" aria-label="close"> <div class='line'></div> <div class='line'></div> </button> <nav class="main-menu-wrapper"> <ul class="color--orange al-menu-main menu"> <li class="menu-item"> <a href="/" data-drupal-link-system-path="<front>">Home</a> </li> <li class="menu-item"> <a href="/alnylam-rnai" data-drupal-link-system-path="node/5">Alnylam & RNAi</a> </li> <li class="menu-item"> <a href="/congresses-publications" data-drupal-link-system-path="node/51">Congresses & Publications</a> </li> <li class="menu-item"> <a href="/therapeutic-areas" data-drupal-link-system-path="node/47">Therapeutic Areas</a> </li> <li class="menu-item menu-item--active-trail"> <a href="/our-therapies" data-drupal-link-system-path="node/49" class="is-active" aria-current="page">Our Therapies</a> </li> <li class="menu-item"> <a href="/pipeline-clinical-trials" data-drupal-link-system-path="node/50">Pipeline & Clinical Trials</a> </li> <li class="menu-item"> <a href="/connect-with-us" data-drupal-link-system-path="node/24">Connect With Us</a> </li> <li class="menu-item"> <a href="/payer-connect" data-drupal-link-system-path="node/145">Payer Connect</a> </li> </ul> </nav> <nav class="account-menu-wrapper"> <ul class="al-menu-account menu"> <li class="menu-item"> <a href="/user/login" data-drupal-link-system-path="user/login">Log in</a> </li> </ul> </nav> <nav class="signup-menu-wrapper"> <ul class="al-menu-signup menu"> <li class="menu-item"> <a href="/user/register" data-drupal-link-system-path="user/register">Sign Up</a> </li> </ul> </nav> <a href="https://www.alnylam.com" class="ext mobile-menu-logo-link logo-link"> <img class="al-logo" src="/themes/custom/alnylam/assets/dist/images/alnylam-logo-white.svg" alt="Alnylam logo"/> </a> <div class="footer-menu-wrapper"> <nav class="footer-menu-wrapper"> <ul class="al-menu-footer menu"> <li class="menu-item"> <a href="https://alnylampolicies.com/privacy" target="_blank">Privacy Policy</a> </li> <span class="footer-separator"> | </span> <li class="menu-item"> <a href="https://alnylampolicies.com/" target="_blank">Terms of Use</a> </li> </ul> </nav> <span> <span class="footer-separator">|</span> <span> © 2024 Alnylam</span> </span> </div> <div class="legal"> <div class="block-content block-content--type-footer-content block-content--9 block-content--view-mode-full"> <div class="block-content__content"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>This site is intended for US healthcare professionals.<br>© 2024 Alnylam Pharmaceuticals, Inc. All rights reserved.<br>MED-US-ALL-2400001</p></div> </div> </div> </div> </div> </div> </div> </div> </nav> </div> </header> <div class="region region-highlighted"> <div data-drupal-messages-fallback class="hidden"></div> </div> <main role="main container-fluid"> <a id="main-content" tabindex="-1"></a> <div class="row"> <div class="main-nav d-none col-12 col-xl-3 d-xl-block"> <div class="left-side branding"> <a href="/"> <img src="/themes/custom/alnylam/assets/dist/images/rnaiscience-logo.svg" alt="RNAiScience logo"/> </a> </div> <aside class="layout-sidebar-first" role="complementary"> <div class="top-wrapper"> <nav class="main-menu-wrapper"> <ul class="color--orange al-menu-main menu"> <li class="menu-item"> <a href="/" data-drupal-link-system-path="<front>">Home</a> </li> <li class="menu-item"> <a href="/alnylam-rnai" data-drupal-link-system-path="node/5">Alnylam & RNAi</a> </li> <li class="menu-item"> <a href="/congresses-publications" data-drupal-link-system-path="node/51">Congresses & Publications</a> </li> <li class="menu-item"> <a href="/therapeutic-areas" data-drupal-link-system-path="node/47">Therapeutic Areas</a> </li> <li class="menu-item menu-item--active-trail"> <a href="/our-therapies" data-drupal-link-system-path="node/49" class="is-active" aria-current="page">Our Therapies</a> </li> <li class="menu-item"> <a href="/pipeline-clinical-trials" data-drupal-link-system-path="node/50">Pipeline & Clinical Trials</a> </li> <li class="menu-item"> <a href="/connect-with-us" data-drupal-link-system-path="node/24">Connect With Us</a> </li> <li class="menu-item"> <a href="/payer-connect" data-drupal-link-system-path="node/145">Payer Connect</a> </li> </ul> </nav> </div> <div class="bottom-wrapper"> <a href="https://www.alnylam.com" class="aside-logo-link logo-link"> <img class="al-logo" src="/themes/custom/alnylam/assets/dist/images/Alnylam-logo.svg" alt="Alnylam logo"/> </a> <nav class="footer-menu-wrapper"> <ul class="al-menu-footer menu"> <li class="menu-item"> <a href="https://alnylampolicies.com/privacy" target="_blank">Privacy Policy</a> </li> <span class="footer-separator"> | </span> <li class="menu-item"> <a href="https://alnylampolicies.com/" target="_blank">Terms of Use</a> </li> </ul> </nav> </div> </footer> </aside> </div> <div class="content-wrapper col-12 col-xl-9"> <div class="row"> <nav class="desktop-header col-12 d-none d-xl-block"> <div class="row"> <div class="right-side col-12"> <div class="search"> <div class="block block-alnylam-search block-alnylam-search-form-block"> <form action="/search" method="get" id="views-exposed-form-search-search-block" accept-charset="UTF-8"> <div class="search-block-input"> <label for="edit-query-block" class="visually-hidden">Search</label> <input id="edit-query-block" name="query" placeholder="Search" data-drupal-selector="edit-query-block" type="text" size="30" maxlength="128" class="form-text" autocomplete="false"> <div class="disclaimer"> <div class="block-content block-content--type-basic block-content--6 block-content--view-mode-full"> <div class="block-content__content"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>More than one search term is recommended to yield the most relevant results. By searching, you confirm that your query is unsolicited.</p></div> </div> </div> </div> </div> <div id="edit-actions-block"> <label for="edit-submit-search-block" class="visually-hidden">Submit</label> <input data-drupal-selector="edit-submit-search-block" type="submit" id="edit-submit-search-block" value="" class="button js-form-submit form-submit" aria-label="Submit Search"> </div> </form> </div> </div> <div class="page-links"> <nav class="utility-menu-wrapper"> <ul class="al-menu-utility-menu menu"> <li class="menu-item"> <a href="/connect-with-us#adverse-events-product-complaints" data-drupal-link-system-path="node/24">Report an Adverse Event</a> </li> <li class="menu-item"> <a href="/connect-with-us#msl-locator" data-drupal-link-system-path="node/24">Locate an MSL</a> </li> </ul> </nav> </div> <div class="account-links"> <nav class="account-menu-wrapper"> <ul class="al-menu-account menu"> <li class="menu-item"> <a href="/user/login" data-drupal-link-system-path="user/login">Log in</a> </li> </ul> </nav> <span class="menu-separator">|</span> <nav class="signup-menu-wrapper"> <ul class="al-menu-signup menu"> <li class="menu-item"> <a href="/user/register" data-drupal-link-system-path="user/register">Sign Up</a> </li> </ul> </nav> </div> </div> </div> </nav> </div> <div class="row"> <div class="top-content col-12"> <div class="region region-content"> <div id="block-alnylam-breadcrumbs" class="block block-system block-system-breadcrumb-block"> </div> <div id="block-alnylam-content" class="block block-system block-system-main-block"> <article about="/our-therapies" class="node node--type-generic node--promoted node--view-mode-full"> <div class="node__content"> <h1><span class="field field--name-title field--type-string field--label-hidden">Our Therapies</span> </h1> <div class="meta"> </div> <div class="field field--name-field-modules field--type-entity-reference-revisions field--label-hidden field__items"> <div class="bundle-general-text-section field__item"> <div class="general-text-section paragraph paragraph--type--general-text-section paragraph--view-mode--default color--default"> <div class="clearfix text-formatted field field--name-field-general-text-section-text field--type-text-with-summary field--label-hidden field__item"><p>Explore a breadth of educational, scientific, and clinical resources related to the FDA-approved uses of our therapies.</p><p> </p><p>Can't find what you're looking for? Use the “<a href="/search" data-entity-type="node" data-entity-uuid="9f486a97-df17-4aa0-a594-f7e1b0f74c35" data-entity-substitution="canonical" target="_blank"><strong>Search</strong></a>” feature to submit a keyword search and discover more about our therapies. </p></div> </div> </div> <div class="bundle-full-width-image field__item"> <div class="hero paragraph paragraph--type--full-width-image paragraph--view-mode--default"> <div class="full-width-image"> <div class="image"> <div class="desktop"> <img src="https://www.rnaiscience.com/sites/default/files/styles/webp/public/2024-02/degrading-mrna.png.webp?itok=gMXylahM" alt="Degrading mRNA after cleaving by siRNA loaded RISC" loading="lazy"> </div> <div class="mobile"> <img src="https://www.rnaiscience.com/sites/default/files/styles/webp/public/2024-03/degrading-mrna-mobile.jpg.webp?itok=hnmQKqOC" alt="Degrading mRNA after cleaving by siRNA loaded RISC" loading="lazy"> </div> </div> </div> </div> </div> <div class="bundle-cta field__item"> <div class="paragraph paragraph--type--cta paragraph--view-mode--default"> <div class="field__item"> <a class="cta-btn" href=/our-therapies/givosiran> Givosiran </a> </div> <div class="field__item"> <a class="cta-btn" href=/our-therapies/lumasiran> Lumasiran </a> </div> <div class="field__item"> <a class="cta-btn" href=/our-therapies/patisiran> Patisiran </a> </div> <div class="field__item"> <a class="cta-btn" href=/our-therapies/vutrisiran> Vutrisiran </a> </div> </div> </div> <div class="bundle-general-text-section field__item"> <div class="general-text-section paragraph paragraph--type--general-text-section paragraph--view-mode--default color--default"> <div class="clearfix text-formatted field field--name-field-general-text-section-text field--type-text-with-summary field--label-hidden field__item"><p>For further information, <a href="/connect-with-us" data-entity-type="node" data-entity-uuid="14d16229-75d9-400d-b346-fa3db98f1e49" data-entity-substitution="canonical"><strong>connect with us</strong></a> to contact a Medical Science Liaison or submit a Medical Information request.</p></div> </div> </div> </div> </div> </article> </div> </div> </div> <footer class="desktop-footer col-12 d-none d-xl-block" role="contentinfo"> <div class="block-content block-content--type-footer-content block-content--9 block-content--view-mode-full"> <div class="block-content__content"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>This site is intended for US healthcare professionals.<br>© 2024 Alnylam Pharmaceuticals, Inc. All rights reserved.<br>MED-US-ALL-2400001</p></div> </div> </div> </footer> <footer class="mobile-footer col-12 d-xl-none" role="contentinfo"> <div class="footer-menu-wrapper"> <nav class="footer-menu-wrapper"> <ul class="al-menu-footer menu"> <li class="menu-item"> <a href="https://alnylampolicies.com/privacy" target="_blank">Privacy Policy</a> </li> <span class="footer-separator"> | </span> <li class="menu-item"> <a href="https://alnylampolicies.com/" target="_blank">Terms of Use</a> </li> </ul> </nav> <span> <span class="footer-separator">|</span> <span> © 2024 Alnylam</span> </span> </div> <a href="https://www.alnylam.com" class="ext footer-logo-link logo-link"> <img class="al-logo" src="/themes/custom/alnylam/assets/dist/images/alnylam-logo-white.svg" alt="Alnylam logo"/> </a> </footer> </div> </div> </div> </main> <div class="al-modal external-link-modal non-alnylam inactive"> <div class="modal-wrapper"> <div class="block external-link-modal-block"> <div class="branding"> <img src="/themes/custom/alnylam/assets/dist/images/rnai.svg" alt="RNAI Logo" /> </div> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>You are now leaving this site and being directed to a third-party website that is not maintained by Alnylam. Alnylam does not endorse and is not responsible for content or maintenance of third-party websites.</p><p><a class="button continue-button" href="#">Continue</a><a class="button cancel-button" href="#">Cancel</a></p></div> </div> </div> </div> <div class="al-modal external-link-modal primary-alnylam inactive"> <div class="modal-wrapper"> <div class="block external-link-modal-block"> <div class="branding"> <img src="/themes/custom/alnylam/assets/dist/images/rnai.svg" alt="RNAI Logo" /> </div> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>You are now leaving this site and being directed to another Alnylam website.</p><p><a class="button continue-button" href="#">Continue</a><a class="button cancel-button" href="#">Cancel</a></p></div> </div> </div> </div> <div class="al-modal disclaimer-modal inactive"> <div class="modal-wrapper"> <div class="block disclaimer-modal-block"> <div class="branding"> <img src="/themes/custom/alnylam/assets/dist/images/rnai.svg" alt="RNAI Logo" /> </div> <input class="d-none" type="hidden" value="0" id="disclaimer-value" /> <input class="d-none" type="hidden" value="86400000" id="disclaimer-ttl" /> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Intended for US healthcare professionals. This site is intended to provide balanced, evidence-based information to support scientific exchange. It is not intended to provide medical advice or recommendations for clinical practice.</p><p>Information about investigational therapeutics or investigational uses of approved products does not establish the safety or efficacy of these therapeutics or uses, and there is no guarantee of FDA approval.</p><p>Alnylam does not recommend or suggest use of its products in any manner inconsistent with the approved Prescribing Information.</p><p>Are you a healthcare professional practicing in the United States?</p><p><a class="button hcp-button hcp-button-home" href="#">I confirm I am a US healthcare professional</a></p><p><a class="button hcp-button payer-connect-hcp-button" href="/payer-connect" data-entity-type="node" data-entity-uuid="634243ce-b30b-41b4-82f7-b6ea8dfc7ee4" data-entity-substitution="canonical">I am a US payer or healthcare decision maker</a></p><p><a class="button not-hcp-button" href="#">I am not a US healthcare professional, or a US payer or healthcare decision maker</a></p><p><a class="rep-button" href="#">I am an Alnylam Team Member</a></p></div> </div> </div> </div> <div class="al-modal login-modal inactive"> <div class="block modal-block"> <span class="close">X</span> <div class="modal-title"> <span style="color:#143a66; size:20;">Don't miss out!</span> </div><div class="modal-content"> <p>Sign in to bookmark information and create your personal library</p><p><a class="button" href="/user/login">Log in</a></p><p>Don't have an account? <a href="/user/register"><strong>Sign up</strong></a></p> </div> </div> </div> <div class="modal-background inactive"></div> </div> </div> </div> <script type="application/json" data-drupal-selector="drupal-settings-json">{"path":{"baseUrl":"\/","pathPrefix":"","currentPath":"node\/49","currentPathIsAdmin":false,"isFront":false,"currentLanguage":"en"},"pluralDelimiter":"\u0003","suppressDeprecationErrors":true,"data":{"extlink":{"extTarget":true,"extTargetNoOverride":true,"extNofollow":false,"extNoreferrer":false,"extFollowNoOverride":false,"extClass":"ext","extLabel":"(link is external)","extImgClass":false,"extSubdomains":false,"extExclude":"","extInclude":"","extCssExclude":"","extCssExplicit":"","extAlert":false,"extAlertText":"This link will take you to an external web site. We are not responsible for their content. Click Me!","mailtoClass":"0","mailtoLabel":"(link sends email)","extUseFontAwesome":false,"extIconPlacement":"append","extFaLinkClasses":"fa fa-external-link","extFaMailtoClasses":"fa fa-envelope-o","whitelistedDomains":[]}},"user":{"uid":0,"permissionsHash":"af669ad7e8d0506788efb80fe142a694aa0101197c5f2204180a5ca90a7fdc78"}}</script> <script src="/sites/default/files/js/js_MoQcAcUltphbuDd-mQXlqpZOGyKpdWp2tbTVZ6g5Bho.js?scope=footer&delta=0&language=en&theme=alnylam&include=eJxLzMmrzEnM1U_PyU9KzNHNyUwqSiyq1EmtKMnJzMvWTykqLUjM0YNyAZXZEcA"></script> <script type="module" src="https://d1mu6oy21or6o2.cloudfront.net/v1.0/bundle.js"></script> <mic-mei-component client-id="7755122c-57d1-4b39-80a8-d1fb00e71fc6" class="mic-mei-component-custom" config-name="Alnylam_Web_Component_Template" style="--mei-font-family:Satoshi-Regular; --mei-header-font-size: 2rem; --mei-subheader-font-size: 1.5rem; --mei-preamble-font-size: 0.8em; --mei-footer-font-size: 0.8em; --mei-primary-color:#4e586a; --mei-header-color:#143a66; --mei-subheader-color:#0099dc; --mei-button-color:#005b85; --mei-border: 0.01rem solid #153a64; --mei-button-text-color: #FFF; --mei-icon-closed: url(/themes/custom/alnylam/images/icons/AskMedical.png); --mei-icon-expanded: url(/themes/custom/alnylam/images/icons/AskMedical.png);"></mic-mei-component> </body> </html>